Adrenoleukodystrophy: Effects of dietary restriction of very long chain fatty acids and of administration of carnitine and clofibrate on clinical status and plasma fatty acids

F. R. Brown, M. A.S. van Duyn, A. B. Moser, J. D. Schulman, W. B. Rizzo, R. D. Snyder, J. V. Murphy, S. Kamoshita, C. J. Migeon

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Abstract

Adrenoleukodystrophy (ALD) is an X-linked disorder which involves predominantly the adrenal cortex and the white matter of the central nervous system. Males who are hemizygous for the mutant allele are clinically normal in infancy, but develop signs of progressive cerebral dysfunction and, usually, adrenal failure by the second decade. A variant form of the condition, adrenomyeloneuropathy (AMN) has a more prolonged course and mainly involves the spinal cord and adrenals; because ALD and AMN have occurred in relatives of several families, they likely represent variable expression of the same mutation. A form distinct from ALD and AMN by neonatal onset and rapid deterioration also exists. No therapy is effective in preventing or reversing the clinical course of any form of the disease. We present observations on 7 patients with ALD or AMN who were maintained on a very long chain fatty acid-restricted diet for periods of 4 months to 2 yr. A preliminary evaluation of the effect of clofibrate and carnitine on plasma levels of very long chain fatty acids was also conducted.

Original languageEnglish (US)
Pages (from-to)164-172
Number of pages9
JournalJohns Hopkins Medical Journal
Volume151
Issue number4
StatePublished - Dec 1 1982

ASJC Scopus subject areas

  • Medicine(all)

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    Brown, F. R., van Duyn, M. A. S., Moser, A. B., Schulman, J. D., Rizzo, W. B., Snyder, R. D., Murphy, J. V., Kamoshita, S., & Migeon, C. J. (1982). Adrenoleukodystrophy: Effects of dietary restriction of very long chain fatty acids and of administration of carnitine and clofibrate on clinical status and plasma fatty acids. Johns Hopkins Medical Journal, 151(4), 164-172.